A Prospective Observational Study of Foam Sclerotherapy .

A Prospective Observational Study of Foam Sclerotherapy for the Treatment of Symptomatic Kidney or Liver Cysts in Patients With Autosomal Dominant Polycystic Kidney and Liver Disease.

An observational prospective study to determine the impact of foam sclerotherapy of large, dominant kidney/liver cysts on quality of life outcomes and kidney/liver cyst volumes at up to 12 months of follow-up in patients with autosomal dominant polycystic kidney disease (ADPKD) and autosomal dominant polycystic liver disease (ADPLD).

Study Overview

• Status: Recruiting
• Conditions: Autosomal Dominant Polycystic Kidney; Autosomal Dominant Polycystic Liver Disease; Renal Cyst; Hepatic Cyst

Detailed Description

Cyst burden is an important determinant of outcomes in both autosomal dominant polycystic kidney disease (ADPKD) (1, 2) and autosomal dominant polycystic liver disease (ADPLD) (3, 4). Furthermore, mass symptoms (from liver and kidney volume) greatly impact upon quality of life in patients with severe disease. Cyst volume increases exponentially with age and results in the development of end-stage renal disease and hypertension, compromised quality of life due to compressive symptoms, and predisposes patients to cyst complications such as infection, hemorrhage, rupture, and torsion. Existing percutaneous treatments for cyst burden in ADPKD and ADPLD include cyst aspiration with or without sclerotherapy. Although frequently effective in the short-term, recurrence rates and the need for repeat procedures are high after these procedures (5, 6). Extrarenal disease (primarily liver disease) is the most important aspect of disease burden to ADPKD patients (7), and there are few effective treatments.

Foam sclerotherapy (FS) with 3% Sodium Tetradecyl Sulfate (STS) a sclerosing agent. (Sotradecol®; Mylan, Galway, Ireland) is approved by the FDA for the management of varicose veins. While increasing cyst burden significantly compromises quality of life, the impact of FS on patient-reported outcomes has not been evaluated. In collaboration with the Center for Science of Healthcare delivery, we have developed a patient-reported outcome tool for polycystic liver disease capable of detecting symptom burden in individuals with polycystic liver disease that has been approved by the FDA as a patient-reported outcomes tool in research. Furthermore measurement of liver and kidney volumes can be performed in the Polycystic Kidney Disease Imaging Research Core that monitors organ volumes before and after interventions. At this time, patients are interested in procedures that will alleviate and palliate their mass symptoms but desire preliminary information on the procedural efficacy. We aim to report our experience with a new therapeutic advance - FS for the treatment of liver and kidney cysts at Mayo Clinic - and to determine the impact of this procedure on patient-reported quality of life measures and changes in organ volumes.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Cassie Howe, CRC; Phone Number: 507-266-1230; Email: howe.cassie@mayo.edu

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
      • Contact: Marie C Hogan, MD, PhD; Phone Number: 507-266-1963; Email: Hogan.marie@mayo.edu
      • Principal Investigator: Marie C. Hogan, M.D., Ph.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 100 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult subjects with ADPKD or ADPLD and large, symptomatic kidney or liver cysts followed in Nephrology or Hepatology clinics at Mayo Clinic, or de novo referrals to Mayo Clinic, Rochester, MN will be invited to participate.

Description

Inclusion Criteria:

• Age ≥18 years
• Clinical diagnosis of ADPKD or ADPLD
• 1-3 large, non-exophytic liver or kidney cyst
• Compressive symptoms from dominant cyst

Exclusion Criteria:

• Age <18 years
• Absence of ADPKD or ADPLD
• Largest cyst <4cm in all 3 dimensions or largest cyst volume <400cc
• >3 large, non-exophytic liver or kidney cyst(s) ≥4cm in all 3 dimensions (cyst volume ≥400cc)
• Unwilling to comply with study follow-up protocol to 12 ± 1months post-FS
• Lacking capacity to provide informed consent to FS of liver or kidney cysts
• Pregnancy

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Pain Inventory	Measured using self assessed brief pain inventory (short form) questionnaire, using a scale of 0 is no pain and 10 is pain as bad as you can imagine	Baseline, 1 month, 6 month, 12 month
Change in Self Assessed Quality of life	Measured using the linear analogue self assessed questionnaire, using a scale of 0 is as bad as it can be and 10 is as good as it can be	Baseline, 1 month, 6 month, 12 month
Change in health related quality of life	Measured using the self assessed Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36), using a scale that ranges from yes and no or all of the time to none of the time	Baseline, 1 month, 6 month, 12 month
Change in Polycystic Liver Disease Quality of Life	Measured using the self assessed Polycystic Liver Disease Questionnaire, using a scale that ranges from never/not at all to always/a lot	Baseline, 1 month, 6 month, 12 month

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Marie Hogan, MD, PhD, Mayo Clinic

Publications and helpful links

Helpful Links

• Mayo Clinic Clinical Trials

Study record dates

Study Major Dates

• Study Start (Actual): November 6, 2017
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2027

Study Registration Dates

• First Submitted: May 1, 2019
• First Submitted That Met QC Criteria: September 30, 2019
• First Posted (Actual): October 1, 2019

Study Record Updates

• Last Update Posted (Actual): November 30, 2023
• Last Update Submitted That Met QC Criteria: November 29, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Kidney Cyst; hepatic cyst; Autosomal Polycystic Liver Disease; Autosomal Polycystic Kidney Disease
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms; Kidney Diseases; Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Joint Diseases; Musculoskeletal Diseases; Muscular Diseases; Pathological Conditions, Anatomical; Musculoskeletal Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Liver Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Arthrogryposis; Cysts
• Other Study ID Numbers: 17-006399

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No